Our weekly newsletter provides you with the latest industry news, developments and regulatory agency updates.
Company Updates
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Regulatory Updates
European shortages monitoring platform enhances tracking of shortages across EU
On 28th November, the European Medicines Agency (EMA) announced that the European Shortages Monitoring Platform (ESMP) has gone live with a core set of functionalities. Using this first version of the ESMP, marketing authorisation holders (MAHs) can now submit data to routinely report shortages of centrally authorised medicines. This marks the start of a transition period that will end on 2 February 2025, when the use of the platform becomes mandatory.
Final call issued to comply with Windsor Framework arrangements for medicines
On 29th November, the Medicines and Healthcare products Regulatory Agency (MHRA), while recognising industry is already showing a very high level of readiness, issued a final call to marketing authorisation holders to take immediate action in preparation for the December 2024 deadline. From 1st January 2025, the MHRA will be responsible for licensing all medicines on the market in Northern Ireland under a UK-wide licence, and all medicines for the UK market must carry a ‘UK Only’ label or sticker to show that they are only for sale within the UK and not in countries inside the EU.
MHRA guidance updates
- Good laboratory practice (GLP) for safety tests on chemicals. On 3rd December, updated the list of UK GLP compliance monitoring programme members. Click to view source
FDA guidance updates
- Use of Circulating Tumour Deoxyribonucleic Acid for Early-Stage Solid Tumour Drug Development; Guidance for Industry; Availability. On 27th November, the FDA published final guidance to help sponsors planning to use circulating cell-free plasma-derived tumour DNA (ctDNA) as a biomarker in cancer clinical trials conducted under an investigational new drug application (IND) and/or to support marketing approval of drugs and biological products for treating solid tumour malignancies in the early-stage (curative-intent) setting.
- Marketing Submission Recommendations for a Predetermined Change Control Plan for Artificial Intelligence-enabled Device Software Functions. On 4th December, final guidance was published with recommendations for predetermined change control plans (PCCPs) tailored to artificial intelligence (AI)-enabled devices. The recommendations in this guidance are intended to support iterative improvement through modifications to AI-enabled devices while continuing to provide a reasonable assurance of device safety and effectiveness.
- CVM GFI #49 Evaluating Target Animal Safety and Effectiveness of Antibacterial New Animal Drugs for Bovine Mastitis. On 4th December, draft guidance was published to provide target animal safety and effectiveness study design considerations and recommendations for sponsors of bovine mastitis drug products with antibacterial activity that are administered by intramammary infusion.
MHRA trials five innovative AI technologies as part of pilot program to transform regulatory approach
On 4th December, the MHRA revealed the selection of AI-powered medical devices for the AI Airlock pilot scheme, which aims to investigate how AI medical devices can be regulated to provide fast and safe access for the NHS and patients. The AI Airlock functions as a “sandbox,” allowing manufacturers to collect evidence under the supervision of the MHRA in a controlled setting. The results will guide future phases of the AI Airlock in the short term and shape MHRA guidelines and policies in the long term while also examining any limitations of existing approaches to demonstrating regulatory compliance.
Industry Updates
MHRA approves new diagnostic agent for signs of cognitive impairment for Alzheimer’s disease
On 27th November, the MHRA approved flortaucipir (Tauvid), a radiopharmaceutical product for adults with memory issues. It is used to assist in diagnosing conditions like Alzheimer’s disease by helping doctors detect abnormal tau protein in the brain through a PET (Positron Emission Tomography) scan. The agency will continue to monitor the safety of flortaucipir, and patients are encouraged to report any side effects to the Yellow Card scheme.
FDA explores improved communication of drug safety information for lactating patients in new study
On 26th November, the FDA published a Spotlight on CDER Science featuring a collaboration between the Center for Drug Evaluation and Research (CDER), the Center for Biologics Evaluation and Research and outside researchers to conduct a study to explore if adding a concise summary of risk information would more succinctly communicate drug safety information to healthcare providers (HCPs), and, in combination with the Lactation subsection narrative included in prescribing information (PI), support HCP clinical decision-making for lactating patients. This research advances CDER’s efforts to understand how HCPs interpret the Lactation subsection of the PI and how the PI can be improved to better communicate information to HCPs about the safety of prescription drugs used during lactation.
BridgeBio secures FDA approval for heart disorder drug
On 22nd November, BridgeBio Pharma, a biopharmaceutical company focused on genetic diseases, announced that the FDA has approved Attruby™ (acoramidis), an orally administered near-complete (≥90%) stabiliser of Transthyretin (TTR) for the treatment of adults with ATTR-CM to reduce cardiovascular death and cardiovascular-related hospitalisation. The approval is based on positive results seen in the ATTRibute-CM Phase 3 study, where Attruby significantly reduced death and cardiovascular-related hospitalisation, and improved quality of life.
OGT launches updated genetic profiling panel for CLL
On 2nd December, OGT announced the launch of its updated NGS SureSeq™ CLL + CNV V3 Panel, which further incorporates the company’s highly sensitive proprietary bait designs to offer more expansive coverage of disease-associated genes and enhanced CNV detection. This new panel now offers users more comprehensive genetic profiling for chronic lymphocytic leukaemia (CLL) samples and allows for better tumour content estimation and reducing inter-run variability by using internal reference DNA. The SureSeq CLL + CNV V3 Panel aims to help researchers make informed decisions about their samples, improving confidence in disease classification and enhancing the understanding of CLL progression.
Orbit Discovery and Evergreen Theragnostics extend research collaboration to advance targeted therapeutics development
On 3rd December, Orbit Discovery, a peptide discovery service provider, and Evergreen Theragnostics, a radiopharmaceutical company, announced an extension of their research collaboration to discover novel targeting peptides for radiopharmaceutical delivery. This collaboration expands upon a previous agreement signed between Orbit and Evergreen in April 2023, whereby technology synergies enabled the identification of high-affinity peptide ligands optimised for precision therapeutic applications. The extended collaboration will utilise Orbit Discovery’s proprietary screening platform to identify and optimise peptide candidates alongside Evergreen Theragnostics’ expertise in radiopharmaceutical development and clinical translation.
Everest Medicines reports positive results from Phase 1b/2a trial of EVER001 for primary membranous nephropathy
On 4th December, Everest Medicines, a biopharmaceutical company, revealed promising results from the Phase 1b/2a clinical trial of EVER001, a Bruton’s tyrosine kinase (BTK) inhibitor, for treating primary membranous nephropathy (pMN). Compared to covalent irreversible BTK inhibitors, EVER001 offers improved selectivity while maintaining high potency, thereby potentially avoiding many of the side effects associated with earlier-generation BTK inhibitors
Antag secures €80 Million in Series A Funding to advance obesity treatment pipeline
On 4th December, Antag Therapeutics, a biopharmaceutical company developing innovative treatments for obesity, announced the completion of an €80 million Series A financing. The round was led by Versant Ventures, with participation from Novo Holdings, SR One, Dawn Biopharma (a KKR-controlled platform), Pictet, Longview Ventures (an affiliate of Broadview Ventures), and the Export and Investment Fund of Denmark (EIFO). The funds will support the clinical development of AT-7687, a novel once-weekly subcutaneous antagonist of the Glucose Dependent Insulinotropic Polypeptide Receptor (GIPR), and further advance Antag’s pipeline of monthly injectable therapies.
GSK and Zhifei revise strategic vaccine collaboration in China
On 5th December, GSK plc announced that it has reached an agreement with Chongqing Zhifei Biological Products, Ltd. (Zhifei) to amend the terms under which Zhifei will commercialise GSK’s shingles vaccine, Shingrix, in mainland China. The updated agreement extends the original 3-year period (2024-2026) during which Zhifei holds exclusive rights to import, distribute, and co-promote the vaccine, adding 8 years, extending through 2034, with revised volume expectations. As part of the new terms, Zhifei will also exclusively collaborate with GSK to explore a potential partnership for the commercialisation of a respiratory syncytial virus (RSV) vaccine in mainland China, contingent upon the vaccine’s regulatory approval.
Issue Number: WN00191
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